Nature Communications (Jun 2021)
A deep learning approach to identify gene targets of a therapeutic for human splicing disorders
- Dadi Gao,
- Elisabetta Morini,
- Monica Salani,
- Aram J. Krauson,
- Anil Chekuri,
- Neeraj Sharma,
- Ashok Ragavendran,
- Serkan Erdin,
- Emily M. Logan,
- Wencheng Li,
- Amal Dakka,
- Jana Narasimhan,
- Xin Zhao,
- Nikolai Naryshkin,
- Christopher R. Trotta,
- Kerstin A. Effenberger,
- Matthew G. Woll,
- Vijayalakshmi Gabbeta,
- Gary Karp,
- Yong Yu,
- Graham Johnson,
- William D. Paquette,
- Garry R. Cutting,
- Michael E. Talkowski,
- Susan A. Slaugenhaupt
Affiliations
- Dadi Gao
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Elisabetta Morini
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Monica Salani
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Aram J. Krauson
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Anil Chekuri
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Neeraj Sharma
- McKusick-Nathans Department of Genetic Medicine, Johns Hopkins University School of Medicine
- Ashok Ragavendran
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Serkan Erdin
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Emily M. Logan
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Wencheng Li
- PTC Therapeutics, Inc.
- Amal Dakka
- PTC Therapeutics, Inc.
- Jana Narasimhan
- PTC Therapeutics, Inc.
- Xin Zhao
- PTC Therapeutics, Inc.
- Nikolai Naryshkin
- PTC Therapeutics, Inc.
- Christopher R. Trotta
- PTC Therapeutics, Inc.
- Kerstin A. Effenberger
- PTC Therapeutics, Inc.
- Matthew G. Woll
- PTC Therapeutics, Inc.
- Vijayalakshmi Gabbeta
- PTC Therapeutics, Inc.
- Gary Karp
- PTC Therapeutics, Inc.
- Yong Yu
- PTC Therapeutics, Inc.
- Graham Johnson
- NuPharmAdvise LLC
- William D. Paquette
- Albany Molecular Research Inc.
- Garry R. Cutting
- McKusick-Nathans Department of Genetic Medicine, Johns Hopkins University School of Medicine
- Michael E. Talkowski
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- Susan A. Slaugenhaupt
- Center for Genomic Medicine, Massachusetts General Hospital Research Institute
- DOI
- https://doi.org/10.1038/s41467-021-23663-2
- Journal volume & issue
-
Vol. 12,
no. 1
pp. 1 – 15
Abstract
Drugs that modify RNA splicing are promising treatments for many genetic diseases. Here the authors show that deep learning strategies can predict drug targets, strongly supporting the use of in silico approaches to expand the therapeutic potential of drugs that modulate RNA splicing.
